Key result
Preclinical gene therapy for cardiomyopathies shows promise for improving cardiac function and reducing fibrosis.
Why the study?
Cardiomyopathies are a heterogeneous group of heart muscle diseases, and novel therapeutic strategies are emerging to target their genetic basis.
Do gene therapy strategies improve cardiac function and disease phenotype in models of cardiomyopathies?
Do gene therapy strategies improve cardiac function and disease phenotype in models of cardiomyopathies?
Gene therapy strategies show promising outcomes in preclinical models of cardiomyopathies, but significant translational and ethical challenges remain before clinical application.
Gene therapy for cardiomyopathies should not yet change practice; leaves open translation of preclinical benefits to clinical trials.
Background: Cardiomyopathies represent a heterogeneous group of heart muscle diseases with diverse etiologies. With the advent of molecular biology and genetic engineering, novel therapeutic strategies are being developed and explored to target the genetic basis of these diseases. Methods: A scoping review was performed of studies published between 2017 and 2023, exploring gene-based therapeutic interventions for various forms of cardiomyopathies. Literature was selected from a wide range of international databases, including PubMed, EMBASE, and Web of Science, with selection criteria focused on the application of gene therapies in preclinical and clinical studies. Findings: A total of 9 studies were included in the review, revealing various gene therapy strategies, including antisense therapies, gene editing, miRNA manipulation, and the use of gene therapy vectors for targeted treatment. Although most of the studies were conducted in preclinical models, they exhibited promising outcomes such as improved cardiac function, reduced fibrosis, and amelioration of disease phenotype. The review also highlighted the considerable heterogeneity among studies in terms of the disease models used, targeted genes, and measured outcomes, indicating the complexity of the field. Conclusion: This scoping review presents an overview of the emerging gene therapy strategies for cardiomyopathies, revealing promising outcomes in preclinical models. However, several challenges remain, including the translation of findings from animal models to humans, ethical considerations, and potential disparities in access to these treatments. Future research should focus on addressing these challenges to pave the way for innovative, efficacious, and accessible therapies for patients with cardiomyopathies.
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Gopal et al. (2023) conducted a review in Cardiomyopathies (n=9). Gene therapy strategies (antisense therapies, gene editing, miRNA manipulation, gene therapy vectors) was evaluated. A scoping review of 9 studies on gene therapy for cardiomyopathies revealed promising preclinical outcomes, including improved cardiac function and reduced fibrosis.
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